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从高吞吐量系统到临床规模的加速腺相关病毒上游过程发展

Angela Andaluz¹, Brittany Monteverde¹, Kevin Vera¹

¹BioProcess Sciences, Pharma Services, Viral Vector Services, Thermo Fisher Scientific, Plainville, Massachusetts, USA.

Biotechnology and bioengineering

|August 21, 2025

概括

这项研究介绍了一种简化基因疗法制造过程,用于产生腺相关病毒 (AAV). 它详细介绍了快速发展和扩展, 降低成本和杂质, 以提供高效的基因疗法.

科学领域:

生物技术
基因治疗制造业
过程开发

背景情况:

腺相关病毒 (AAV) 是基因治疗中的关键传递载体.
目前的AAV制造面临的挑战包括商品的高成本 (COG),可扩展性以及空体等杂质.

研究的目的:

为开发和扩大AAV上游生产提供简化方法.
为了优化生产力,减少杂质,降低每剂COG.
为了证明从测试台扩展到超过1000升的商业容量.

主要方法:

用于AAV生产的三重转染.
采用像AMBR®15系统这样的高吞吐量技术来快速开发过程.
详细的直接扩展方法到2000升一次性生物反应器.

主要成果:

在不到2个月的时间内实现了快速的上游工艺开发.
成功优化了AAV生产以提高生产率和减少杂质.
展示了扩展AAV制造的简化过程.

结论:

这种简化方法可以实现高效且可扩展的AAV制造.
高通量技术加速了过程开发和优化.
这种方法解决了AAV生产的关键问题,为具有成本效益的基因疗法铺平了道路.

关键词:

腺相关病毒良好的降低成本基因疗法高吞吐量发展扩大规模转染

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